Zydus Lifesciences Limited, a global, discovery-driven pharmaceutical company, announced that the U.S. Food and Drug Administration (US FDA) has granted Orphan Drug Designation (ODD) to its investigational drug Desidustat for the treatment of beta-thalassemia.
The designation, issued by the FDA’s Office of Orphan Products Development, supports therapies aimed at treating rare diseases affecting fewer than 200,000 people in the United States.
Commenting on the development, Dr. Sharvil Patel, Managing Director, Zydus Lifesciences, said:
“This Orphan Drug Designation from the US FDA underlines the urgent medical need to develop Desidustat to address beta-thalassemia.”
Beta-thalassemia is a hereditary blood disorder characterized by low haemoglobin levels, leading to chronic fatigue, weakness, and potential organ complications. Current treatments typically involve lifelong blood transfusions and iron chelation therapy to manage iron overload.
Desidustat is an oral hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI) that has demonstrated potential to increase haemoglobin and red blood cell counts. Preclinical studies in beta-thalassemic mice showed a rise in haemoglobin and RBC levels following Desidustat treatment.
The Orphan Drug Designation provides Zydus with several development incentives, including tax credits for clinical testing, waiver of FDA user fees, and up to seven years of market exclusivity upon approval in the United States.